Study BRT-DA01-301 is a Phase 3 multicenter, randomized, sham surgery-controlled, double-blind study to assess efficacy and safety of bemdaneprocel in approximately 102 adults with Parkinson's Disease (PD).
The BRT-DA01-301 study is a Phase 3, multicenter, randomized, sham surgery-controlled, double-blind study involving approximately 102 participants with Parkinson's Disease (PD). Participants will be randomized in a 2:1 ratio to either receive bemdaneprocel or undergo sham surgery. The study includes an immunosuppression regimen and placebo equivalents to maintain blinding. The primary objective is to evaluate the efficacy of bemdaneprocel on motor symptoms in participants with PD. The secondary objective is to evaluate the effects of bemdaneprocel on Motor function, Quality of life, Non-motor symptoms of PD, Disease severity, and Use of PD medications or therapies compared with participants who undergo sham surgery. Participants will be followed for at least 18 months in the double-blind period and up to five years if they receive bemdaneprocel.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
102
Investigational cell therapy comprising midbrain dopaminergic neuron progenitors derived from human embryonic stem cells
Sham surgery will be performed on Day 0
Change from baseline in PD diary measure of ON-time without troublesome dyskinesia, adjusted for a 16-hour waking day
Time frame: From baseline to Week 78
Change from baseline in PD diary measure of OFF-time, adjusted for a 16-hour waking day
Time frame: From baseline to Week 78
Change from baseline in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III score in the off-medication state. Part III score can range from 0 to 132, with lower scores being better.
Time frame: From baseline to Week 78
Change from baseline in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II score. Part II score can range from 0 to 52, with lower scores being better.
Time frame: From baseline to Week 78
Change from baseline in Parkinson's Disease Questionnaire-39 (PDQ-39) summary index. Summary index score can range from 0 to 100, with lower scores being better.
Time frame: From baseline to Week 78
Incidence and severity of treatment-emergent adverse events
Time frame: From baseline to month 60
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